Pharmacokinetics in End Stage Renal Disease Patients

Phase 1

Completed

• Conditions: Healthy Volunteers; Renal Failure
• Interventions: Drug: firibastat

• Registration Number: NCT04792333
• Lead Sponsor: Quantum Genomics SA

Brief Summary

The study is a multicentre, open label, phase I, two arms study to compare pharmacokinetic of firibastat after a single oral dose of firibastat 500 mg in fourteen healthy male volunteers and in fourteen End Stage Renal Disease (ESRD) patients not yet in dialysis.

Detailed Description

It is planned to enroll a total of 28 subjects to receive a single oral dose of investigational medicinal product (IMP): 14 ESRD patients will be enrolled in three Hungarian centres (1st arm) and 14 Healthy volunteers will be enrolled by Eurofins Optimed in Gières, France (2nd arm).

Subjects will be screened for eligibility to participate in the study up to 21 days prior to the first administration. For both arm, subjects will be admitted into the Clinical Research Unit (CRU) on Day -3. On the morning of Day 1, subjects will receive a single 500 mg oral dose of firibastat following an overnight fast (i.e., at least 10 hours). Subjects will be confined to the CRU until discharge on Day 3 with PK blood sample draws for measurement of firibastat and its main metabolites being taken throughout the confinement.

A follow-up post study visit will occur on Day 10 post-dose to ensure the ongoing wellbeing of the subjects.

Study Timeline

• Study Start: 2019-09-17
• Study First Submitted: 2019-12-26
• Primary Completion: 2020-07-10
• Study Completion: 2020-07-10
• Status Verified: 2021-03
• Study First Submitted QC: 2021-03-09
• Last Update Submitted: 2021-03-09
• Study First Posted: 2021-03-10
• Last Update Posted: 2021-03-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 28

Inclusion Criteria

• Male subjects, aged 18 to 55 years inclusive;
• Non-smoker subject or smoker of not more than 5 cigarettes a day;

Exclusion Criteria

• Positive Hepatitis B surface (HBs) antigen or anti Hepatitis C Virus (HCV) antibody, or positive results for Human Immunodeficiency Virus (HIV) 1 or 2 tests);
• History or presence of drug or alcohol abuse (alcohol consumption > 40 grams/day);

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Treatment	firibastat	500 mg (2 capsules of 250 mg)

Primary Outcome Measures

Name	Time	Method
Firibastat and metabolites Concentrations	Day 1 to Day 3	The plasma concentration data for firibastat (QGC001), EC33 and QGC515, will be analysed at each time measurement (predose, 0, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48 hours post-dose)

Secondary Outcome Measures

Name	Time	Method
Heart Rate (bpm)	Day 1 to Day 3	number of beats per minute
Hematology blood sample laboratory tests aggregated as number of patients outside ranges	Day 1 to Day 3	Hemoglobin, Hematocrit, MCH, MCHC, MCV, MPV, RBC (erythrocytes), WBC (Leucocytes), Differential Count (Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Large Unstained cells), Platelets.
Systolic and Diastolic Blood Pressure (mmHg)	Day 1 to Day 3	Systolic and Diastolic blood pressure
Urinalysis tests aggregated as number of patients outside ranges	Day 1 to Day 3	pH, ketone bodies, proteins, glucose, occult blood, blood leukocytes, nitrite, bilirubin, urobilinogen, density.
Biochemistry blood sample laboratory tests aggregated as number of patients outside ranges	Day 1 to Day 3	Albumin, Alkaline Phosphatase, ALT, Amylase, AST, Bicarbonate, Bilirubin (direct, indirect and total), Calcium, Chloride, Cholesterol, Creatinine Kinase, Creatinine, gammaGT, HDLcholesterol, LDH, LDL - cholesterol, Lipase, Magnesium, Phosphate, Potassium, Protein total, Sodium, Triglycerides, Urea, Uric Acid, Glucose.
Hemostasis blood sample laboratory tests aggregated as number of patients outside ranges	Day 1 to Day 3	INR, PT, aPTT

Trial Locations

Locations (1)

Eurofins Optimed; 🇫🇷 Gières, France